AveXis renamed Novartis Gene Therapies, signifying the growing importance of gene therapy to Novartis corporate strategy

Novartis, Sep 02, 2020

Novartis has renamed the previously acquired AveXis to Novartis Gene Therapies. The change signifies the growing importance of gene therapy to building a leading, focused medicines company with advanced therapy platforms.

The change to Novartis Gene Therapies is the natural evolution as the company scales up to deliver Zolgensma globally and expand its reach via a robust pipeline of AAV-based gene therapies for rare genetic diseases including investigational treatments for Rett syndrome, a genetic form of amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia. Novartis Gene Therapies also establishes a seamless global presence for Zolgensma and the gene therapies to come. Instead of alternating between the AveXis and Novartis umbrella brands by market, the company comes together under one banner as a unified entity.

Reata Announces the Presentation of the Pivotal MOXIe Part 2 Study of Omaveloxolone in Friedreich’s Ataxia at the American Academy of Neurology

September 03, 2020 06:45 ET | Source: Reata Pharmaceuticals, Inc.

PLANO, Texas, Sept. 03, 2020 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” or “we”), a clinical-stage biopharmaceutical company, today announced the forthcoming presentation of efficacy and safety results from the pivotal MOXIe Part 2 study, a randomized, double-blind, placebo-controlled trial of omaveloxolone in Friedreich’s ataxia.

The presentation will take place on September 24, 2020 as part of the 2020 Emerging Science presentations hosted by the American Academy of Neurology (AAN). David Lynch, M.D., Ph.D., will present the data. Dr. Lynch is an attending physician at the Children’s Hospital of Philadelphia (CHOP), professor of neurology at the Perelman School of Medicine at the University of Pennsylvania, and the principal investigator of the MOXIe study.

Separately, Dr. Lynch will present the results of the MOXIe Part 2 study at the FARA 2020 Biomarker & Clinical Endpoint Meetin

High levels of frataxin overexpression leads to mitochondrial and cardiac toxicity in mouse models

Brahim Belbellaa, Laurence Reutenauer, Nadia Messaddeq, Laurent Monassier, Hélène Puccio; Molecular Therapy - Methods & Clinical Development, 2020, doi:10.1016/j.omtm.2020.08.018.

this study underlines the need, during the development of gene therapy approaches, to consider appropriately vector expression level, long term safety and biomarkers to monitor such events.